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. 2022 Apr 4;17(4):e0266353.
doi: 10.1371/journal.pone.0266353. eCollection 2022.

Guidance for pediatric use in prescription information for novel medicinal products in the EU and the US

Helle Christiansen  1 , Marie L De Bruin  1   2 , Sven Frokjaer  1 , Christine E Hallgreen  1
Affiliations

Guidance for pediatric use in prescription information for novel medicinal products in the EU and the US

Helle Christiansen et al. PLoS One. .

Abstract

Pediatric legislations in the European Union (EU) and the United States (US) have increased medicines approved for use in the pediatric population. Despite many similarities between these frameworks, the EU Paediatric Regulation more often provides regulators with a mandate to require pediatric drug development for novel medicinal products compared to US regulators. If used, this could give rise to differences in the guidance for pediatric use provided for clinicians in the two regions. However, the level of discordance in the guidance for pediatric use between the two regions is unknown. This cross-sectional study compares guidance for pediatric use in the EU Summary of Product Characteristics (SmPC) and the US Prescription Information (USPI) on the level of indications granted for novel medicinal products approved after the pediatric legislations came in to force in both regions. For all indications granted as of March 2020 for novel medicinal products approved in both regions between 2010 and 2018, we compared the guidance for pediatric use in the EU SmPC and the USPI. The guidance for pediatric use differed for 18% (61/348) of the listed indications covering 21% (45/217) of the products, but without the guidance being contradictory. Where guidance differed, an equal share was observed for indications with a higher level of information for pediatric use in one region over the other (49% (30/61) in the US; 51% (31/61) in the EU). The discrepancies in pediatric information could be explained by differences in regulations for 21% (13/61) of the indications. Only a few conditions and diseases (EU n = 4; US n = 1) were observed to cover potential pediatric use outside the approved adult indication. Although the EU Paediatric Regulation more often provides regulators a mandate for requiring pediatric drug development as compared to the US PREA, this was not reflected in the prescription information approved by the two regulatory authorities.

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Conflict of interest statement

The authors have declared that no competing interests exist.

Figures

Fig 1
Fig 1. Categorization and hierarchical grouping of pediatric information.
A) Tiered categorization of pediatric information in the SmPC and the USPI and B) hierarchical grouping of the categorized pediatric information to provide guidance for pediatric use.
Fig 2
Fig 2. Selection of cohort.
Flowchart of selection of the new active substances (products) included in the study and the indications for analysis.
See this image and copyright information in PMC

References

    1. FDA. Guidance for Industry and Review Staff Pediatric Information Incorporated Into Human Prescription Drug and Biological Products Labeling. 2019 [Accessed February 2022]. Available from https://www.fda.gov/regulatory-information/search-fda-guidance-documents...
    1. European Commission. Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004. [Accessed February 2022]. Available from: https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32006R1901...
    1. FDA. Pediatric Research Equity Act of 2003. [Accessed February 2022]. Available from: https://www.congress.gov/108/plaws/publ155/PLAW-108publ155.pdf
    1. FDA. Best Pharmaceuticals for Children Act of 2002. [Accessed February 2022]. Available from: https://www.congress.gov/107/plaws/publ109/PLAW-107publ109.pdf
    1. European Commission. A guideline on Summary of Product Characteristics (SmPC). 2009 [Accessed February 2022]. Available from: https://ec.europa.eu/health/system/files/2016-11/smpc_guideline_rev2_en_...

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